CRISPR-modified hematopoietic stem cells

LifeStem's CRISPR-modified hematopoietic stem cell therapy is an experimental ex vivo gene-edited cell therapy developed for the treatment of Beta-Thalassemia, specifically targeting the IVS-1-110 mutation. The therapy utilizes CRISPR/Cas9 technology to perform precise gene correction of the HBB gene in autologous hematopoietic stem cells (HSCs) harvested from the patient. By correcting the splicing defect caused by the IVS-1-110 mutation, the therapy aims to restore the production of functional beta-globin protein. Once the cells are edited and validated, they are re-infused into the patient, where they are intended to engraft in the bone marrow and produce healthy red blood cells, potentially providing a curative one-time treatment for this specific genetic variant of Beta-Thalassemia.

Other names

CRISPR HSCsLifeStem CRISPR-HSC therapyex vivo CRISPR gene editing for Beta-Thalassemia